clinical-trial
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Going boldly: First person treated in Phase 1 clinical trial by Alnylam Pharmaceuticals
A new Phase 1 clinical trial for the huntingtin-lowering drug ALN-HTT02 was initiated this week with the first dose given. Read on to learn details about the trial and how it compares to other ongoing huntingtin-lowering trials in the clinic.
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The dust has settled: Sage’s dalzanemdor won’t advance for cognitive impairment
Changes in thinking, learning, and memory are some of the most distressing and impactful in Huntington’s disease. Sage Therapeutics was advancing their drug dalzanemdor to treat these symptoms, but unfortunately this program will be halted.
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Bringing HD Treatments to Market: The Role of Regulatory Oversight
Moving drugs from the lab bench to pharmacy shelves is no small task. In this article we delve into the role regulatory oversight plays in clinical trials and the approval of medicines for Huntington’s disease.
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Hope vs. hype: seeking truth in recent Prilenia headlines
Although pridopidine has suffered four negative trials for HD, the message from Prilenia continues to be positive. What is hope and what is hype in this sixteen-year quest for regulatory approval?
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Blue skies for Skyhawk: Positive news from Phase 1 trial for SKY-0515
There’s more good news in the forecast in the Huntington’s disease therapeutic space as we receive positive results from Skyhawk Therapeutics about their small molecule SKY-0515 that lowers huntingtin and targets somatic expansion.
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Buckle in: Gene therapy AMT-130 appears to slow down signs of Huntington’s disease in Phase I/II clinical trial
More good news for the HD community from uniQure, whose one-and-done gene therapy appears safe and shows hints of slowing down signs and symptoms of the disease
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Positive news from Wave Life Sciences SELECT-HD trial
The outcomes of the SELECT-HD clinical trial are out, with encouraging news for this allele-selective huntingtin lowering therapy
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No pivot needed for PTC-518
PTC Therapeutics shared 12 month data from the PIVOT-HD trial, testing the oral HTT lowering drug PTC-518. While designed to assess safety, they shared encouraging results that the drug showed promising signs for biomarkers and some clinical metrics.
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Huntington's Disease Therapeutics Conference 2024 – Day 3
HDBuzz is back for the last day of the CHDI HD Therapeutics Conference: Thursday February 29th in Palm Springs, California. This article summarizes our real-time updates of the conference in community-friendly language. From genes to medicines The morning session will focus on how human genetics is driving the development of therapeutics. “Genetic modifiers” are genes…
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Putting it in print: GENERATION HD1 study results published
Data from GENERATION HD1, the Phase 3 clinical trial testing the huntingtin-lowering drug tominersen, have just been published in a scientific journal. The trial ended a while back, so why is this an important milestone, and what’s next?